Trikafta for Cystic Fibrosis
(Can-IMPACT CF Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines the long-term effectiveness of a treatment called Trikafta for individuals with cystic fibrosis. Researchers aim to determine the most effective treatments for each person by gathering health data and patient surveys from across Canada. The trial is open to those who are about to start or switch their CFTR modulator therapy (a type of treatment for cystic fibrosis) and are part of the Canadian Cystic Fibrosis Registry.
As a Phase 4 trial, this research provides insights into how the already FDA-approved and proven effective treatment benefits more patients, offering valuable information for those considering participation.
Do I need to stop my current medications for this trial?
The trial does not specify if you need to stop your current medications. It focuses on patients already taking or planning to start CFTR modulator therapy.
What is the safety track record for Trikafta?
Research has shown that Trikafta is generally well-tolerated by people with cystic fibrosis (CF). Safety data from two studies involving 510 patients aged 12 and older indicated that the treatment was effective over 24 weeks. However, some serious risks require attention. Trikafta can harm the liver and, in rare cases, lead to liver failure, resulting in liver transplants and, in some instances, death. Monitoring liver health during treatment is crucial. Overall, most patients manage Trikafta well, but regular check-ups with a doctor are essential.12345
Why are researchers excited about this trial?
Trikafta is unique because it targets the underlying cause of cystic fibrosis by enhancing the function of the CFTR protein, which is defective in people with this condition. Unlike other treatments that mainly manage symptoms, Trikafta works by correcting the dysfunctional protein, potentially improving lung function and reducing respiratory complications. Researchers are excited about Trikafta because it offers hope for a more effective, long-term solution, providing significant enhancements in quality of life for many patients who previously had limited options.
What is the effectiveness track record for Trikafta in treating cystic fibrosis?
Research has shown that Trikafta, a combination of three medicines, significantly improves health in people with cystic fibrosis (CF). In a 24-week study, participants taking Trikafta experienced 63% fewer lung flare-ups compared to those on a placebo. Clinical trials have demonstrated major health benefits, particularly for children with CF. Additionally, about 90% of people with CF can now use this treatment, highlighting its broad effectiveness. This combination therapy effectively targets the root cause of CF, enhancing lung function and overall health. Participants in this trial will begin CFTR modulator therapy, including Trikafta, as part of their clinical care.678910
Who Is on the Research Team?
Jonathan Rayment, MDCM
Principal Investigator
University of British Columbia
Bradley Quon, MD
Principal Investigator
University of British Columbia
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Pre-Therapy
Participants are initiated on CFTR modulator therapy by their treating physician as part of clinical care
Data Collection
Collection of clinical data from routine standard of care, patient-reported outcomes via survey data, and samples for a biobank
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Trikafta
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
All CF people with CF who do not have any known contraindications to CFTR modulator therapy and will be initiated on CFTR modulator therapy by their treating physician as part of clinical care are eligible and will be asked to participate in this study.
Trikafta is already approved in United States, European Union, Canada for the following indications:
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of British Columbia
Lead Sponsor
Citations
Studies and Results | TRIKAFTA® (elexacaftor/tezacaftor ...
Through 24 weeks, the number of pulmonary exacerbations significantly decreased by 63% for people taking TRIKAFTA compared with placebo.
Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in 6
Rationale: Elexacaftor/tezacaftor/ivacaftor (ETI) provided substantial health benefits to children with cystic fibrosis (CF) in clinical trials; there is less ...
LONGITUDE: An observational study of the long-term ...
LONGITUDE is an observational, registry-based cohort study using data from the UK CF Registry to evaluate outcomes of ELX/TEZ/IVA in people aged ≥6 years.
Real-world outcomes and direct care cost before and after ...
With the most recent approval of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), approximately 90% of the CF population is now eligible to ...
Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a ...
In conclusion, this 24-week, phase 3 trial involving 403 patients with cystic fibrosis confirmed the efficacy of triple-combination CFTR ...
Important Safety Information
TRIKAFTA can cause serious liver damage and liver failure. Liver failure leading to transplantation and death has been seen in some people with or without a ...
Safety Profile | TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor ...
Safety data from 510 patients aged 12 years and older with CF with at least one F508del mutation in 2 double‑blind, controlled, Phase 3 trials of 24 weeks ...
TRIKAFTA® (elexacaftor, tezacaftor, and ivacaftor tablets
The safety profile of TRIKAFTA is based on data from 510 CF patients aged 12 years and older in two double-blind, controlled trials of 24 weeks and 4 weeks.
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) ... It is not known if TRIKAFTA is safe and effective in children under 2 years ...
Effectiveness and safety of elexacaftor/tezacaftor/ivacaftor ...
Our data indicate that ETI therapy is well tolerated by children with CF and is effective in improving signs of lung function abnormalities from early ...
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